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FDA Approves Rivaroxaban for 2 New Pediatric Indications

Announced on Dec. 20, the latest approvals for rivaroxaban (Xarelto) mark the 10th and 11th indications for the factor Xa inhibitor in the US.

The US Food and Drug Administration has approved rivaroxaban (Xarelto) for a pair of new pediatric indications, according to a release from the Janssen Pharmaceutical Companies of Johnson & Johnson.

Announced on December 20, the factor Xa inhibitor is now indicated for use in treatment of venous thromboembolism (VTE) and reduction in the risk of recurrent VTE in patients younger than 18 years of age after at least 5 days of initial parenteral anticoagulant treatment and for thromboprophylaxis in children aged 2 years and older with congenital heart disease who have undergone the Fontan procedure.

"Historically, there has been limited guidance and options for healthcare providers on how to help reduce potentially serious, even fatal, blood clots and related events in young children," said Andrew Van Bergen, MD, a pediatric cardiologist at Advocate Children's Hospital, in the aforementioned press release. "We have had to adjust adult doses of standard anticoagulation therapies, which are both burdensome and uncomfortable for patients, and require frequent monitoring. Now that XARELTO® is FDA-approved with weight-based dosing options, either as tablets or liquid formulation, a convenient option is available allowing flexibility to tailor the treatment for my patients. This is a major advancement in antithrombotic care for those patients under the age of 18."

With the approvals marking the 10th and 11th indications for rivaroxaban in the US, the latest approvals were based on data from 2 phase 3 pediatric studies within the EXPLORER program—the EINSTEIN-Jr study, which was the largest study to date evaluating pediatric patients from birth to less 18 years of age with previously diagnosed VTE, and the UNIVERSE study, which was the first clinical trial to examine a DOAC for the prevention of VTE in pediatric patients after recently undergoing the Fontan procedure.

In EINSTEIN-Jr, which was published in The Lancet Haematology, 500 children were enrolled and randomized to bodyweight-adjusted rivaroxaban or standard anticoagulants. The primary efficacy and safety outcomes of the study were symptomatic recurrent venous thromboembolism and major or clinically relevant non-major bleeding, respectively. Results indicated use of rivaroxaban was associated with a similarly low recurrence risk and reduced thrombotic burden as use standard anticoagulants.

The UNIVERSE study, which was published in the Journal of the American Heart Association, was a randomized, multicenter, 2‐part, open‐label study. The first part of the study assessed the pharmacokinetics/pharmacodynamics and safety of rivaroxaban in 12 participants and the second part randomized 100 post-Fontan procedure participants in a 2:1 ratio to open‐label rivaroxaban or acetylsalicylic acid. Results of the study indicated those who received rivaroxaban for thromboprophylaxis had a similar safety profile and fewer thrombotic events than those who received acetylsalicylic acid.

"Today's FDA approval marks two new XARELTO® indications for pediatric patients, an often underrecognized, but especially important patient population," said James List, MD, PhD, Global Therapeutic Area Head, Cardiovascular, Metabolism, & Retina, Janssen Research & Development, LLC. "At Janssen, we are committed to addressing unmet medical needs and the approval of the 10th and 11th indications for XARELTO® underscores its capability in reducing the risk of blood clots and cardiovascular events in patients from young to old and with a variety of conditions."